Pharming Group N.V. is a global biopharmaceutical company headquartered in Leiden, the Netherlands, that develops and commercializes protein replacement therapies and precision medicines for rare diseases. The company operates primarily in the United States, Europe, and other international markets. Its core business is built around a proprietary transgenic technology platform designed to produce recombinant human proteins in the milk of transgenic animals, which allows for the scalable and cost-efficient manufacturing of complex therapeutic proteins. Pharming's lead commercial product is RUCONEST (recombinant human C1 esterase inhibitor), an approved treatment for acute attacks in patients with hereditary angioedema (HAE). The company also commercializes Joenja (leniolisib), an oral small molecule PI3Kδ inhibitor approved for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS). Beyond its commercialized therapies, Pharming is advancing a clinical pipeline focused on expanding the indications for leniolisib to other primary immunodeficiencies and developing new treatments for unserved rare disease patients.
